velaglucerase alfa

Velaglucerase alfa is a recombinant form of the human enzyme glucocerebrosidase, produced using gene activation technology in a human fibroblast cell line. It is indicated for long-term enzyme replacement therapy (ERT) in pediatric and adult patients with type 1 Gaucher disease, a genetic disorder caused by deficiency of the lysosomal enzyme glucocerebrosidase. This deficiency leads to accumulation of glucocerebroside within macrophages, resulting in symptoms such as hepatosplenomegaly, anemia, thrombocytopenia, and bone disease. Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside into glucose and ceramide within lysosomes. The drug’s glycan chains are recognized by mannose receptors on macrophages—the primary cells affected in Gaucher disease—facilitating targeted delivery[1][2][6][7][8]. Developed as an alternative to imiglucerase and taliglucerase alfa, velaglucerase alfa has an amino acid sequence identical to natural human placental glucocerebrosidase[8].

Brand names

VPRIV

Other names

Gene-Activated® human glucocerebrosidaseGene-Activated™ human glucocerebrosidase